Cheng Haihong, Zhou Di, Wang Shouhua, Ding Jun, Ma Fei
Department of Oncology, Xinhua Hospital Affiliated to Shanghai Jiaotong University School of Medicine, Shanghai, China.
Department of General Surgery, Xinhua Hospital Affiliated to Shanghai Jiaotong University School of Medicine, Shanghai, China.
Intractable Rare Dis Res. 2021 May;10(2):146-147. doi: 10.5582/irdr.2021.01038.
Biliary tract cancer (BTC), which includes cholangiocarcinoma and gallbladder carcinoma, is a rare malignancy. Due to its low incidence, drugs treating these diseases are scarce, so they can be considered orphan drugs. The main treatment choice for BTC is chemotherapy with gemcitabine or cisplatin or combined use of both, but patients fail to significantly benefit from established chemotherapy. Advancements in immunotherapy and targeted therapy will shed light on ways to improve clinical outcomes for patients with BTC. In conjunction, more new drugs will come onto the market. This article compares the conditions for development of orphan drugs in different countries and it describes several types of new drugs that were recently approved to treat BTC.
胆道癌（BTC），包括胆管癌和胆囊癌，是一种罕见的恶性肿瘤。由于其发病率低，治疗这些疾病的药物稀缺，因此可被视为孤儿药。BTC的主要治疗选择是使用吉西他滨或顺铂进行化疗，或两者联合使用，但患者未能从既定的化疗中显著获益。免疫疗法和靶向疗法的进展将为改善BTC患者的临床结局提供思路。与此同时，更多新药将上市。本文比较了不同国家孤儿药的开发条件，并介绍了几种最近获批用于治疗BTC的新药类型。
